Is the Orphan Space About to Get Even More Crowded?

As stem cell therapies begin to look towards commercialisation, where will they find the payers?

Last week I was lucky enough to Attend the World Stem Cell and Regenerative Medicine Congress 2014 in London. The stem cells industry is beginning to consider the commercialisation of their therapies, and a theme that ran throughout the conference was the concern that payers would not spend hundreds of thousands of dollars on cell therapies where there already exists comparatively much cheaper options.

Michael May, CEO at Centre for Commercialization of Regenerative medicine (CCRM), warned that payers were unlikely to pay for a new stem cell treatment at $100,000 if there is currently a much much cheaper alternative available. What May emphasised was the amount of headroom a product has: the space between population size and the improved outcome of a treatment compared to existing treatments.

Whilst May expressed hope that the stem cell industry would move towards cheaper therapies which take advantage of the broad medicinal and regenerative uses of stem cells, it’s not hard to see that many developers will be drawn towards orphan indications to take advantage of tax breaks and payers willing to pay.

There is already a number of stem cell treatments that have received orphan designations -Athersys’ MultiStem or ReNeuron’s ReN003- and recently researchers have demonstrated the power of stem cells in mouse MS models by restoring the ability to walk in mice crippled with an autoimmune disease similar to MS.

Whilst of course the continuing development of treatments for untreated rare diseases is a great thing. But the sustainability of the Orphan Drugs Act is looking increasingly dubious down the line. With developers flocking towards orphan drugs, treatments for cancer subsets dominating orphan designations, and now the possibility of a new and fast growing industry entering the fray, the orphan drugs space could be set to get even more crowded.